On October 27, 2021 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that the U.S. Food and Drug Administration (FDA) has reviewed and cleared the Investigational New Drug (IND) application to initiate a Phase 1/1b clinical trial of prexigebersen-A (liposomal Grb2-A or BP1001-A) in patients with solid tumors, including ovarian, endometrial, pancreatic and triple negative breast cancer (Press release, Bio-Path Holdings, OCT 27, 2021, View Source [SID1234591996]).

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"This IND clearance for prexigebersen-A marks an important regulatory milestone for Bio-Path, as we progress our fourth drug candidate into the clinic. Given the formulation enhancements, encouraging pre-clinical data and safety profile we have seen to-date, we are eager to begin this first-in-human study," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "We are particularly encouraged as prexigebersen-A has been shown to enhance chemotherapy efficacy in preclinical solid tumor models and we look forward to confirming these earlier results."

Prexigebersen-A is a modified drug product with the same drug substance as prexigebersen but includes formulation enhancements to produce smaller drug nanoparticles. The goal of this product enhancement was to produce smaller drug nanoparticles that could pass through vasculature pore spaces, thereby enabling release of the drug product into the interior of the tumor to enhance drug effectiveness.

In April 2018, data were presented demonstrating the treatment of solid tumors in gynecologic malignancies with prexigebersen-A at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data showed an eighty-six percent (86%) decrease in tumor burden (p<0.05), and multinodular burden (p<0.01) in the combination prexigebersen-A/paclitaxel group compared with control. In addition, there was no apparent toxicity with mice on combination therapy losing less weight than the control group. This work was subsequently published in the journal Oncotarget in 2020.

"Given the encouraging pre-clinical results that have been generated to-date, we are optimistic that prexigebersen-A may offer much needed respite to patients suffering with solid tumors who face limited treatment options," said Jorge Cortes, M.D., Director of the Georgia Cancer Center and Chairman of the Bio-Path Scientific Advisory Board.

The Phase 1/1b clinical trial is anticipated to be conducted at several leading cancer centers in the United States, including The University of Texas MD Anderson Cancer Center and the Mary Crowley Research Center. Eventually, we expect to have six sites to conduct the clinical trial. Initially, a total of six evaluable patients are scheduled to be treated with prexigebersen-A monotherapy in a standard 3+3 design, with a starting dose of 60 mg/m2. The approved treatment cycle is two doses per week over four weeks, resulting in eight doses administered over twenty-eight days. The Phase 1b portion of the study will commence after successful completion of prexigebersen-A monotherapy cohorts and will assess the safety and efficacy of prexigebersen-A in combination with paclitaxel in patients with recurrent ovarian or endometrial tumors.

The IND review process was performed by the FDA’s Division of Oncology 1, Office of Oncologic Diseases and involved a comprehensive review of data submitted by the Company covering pre-clinical studies, safety, chemistry, manufacturing and controls, and the protocol for the Phase 1/1b clinical trial.